NIH study in NEJM shows Novartis drug eltrombopag as first-line therapy with standard treatment improves responses in severe aplastic anemia - Seite 3

NIH Study Design
The Phase I-II, non-randomized study is being conducted by the National Heart, Lung and Blood Institute through a Cooperative Research and Development Agreement (CRADA) with Novartis Pharmaceuticals Corporation. The primary analysis included 92 patients with treatment-naïve severe aplastic anemia in three treatment cohorts, and nearly 80% of patients were over the age of 18. Eltrombopag was administered at 150 mg daily for patients 12 years or older, 75 mg daily for those 6 to 11 years, and 2.5 mg/kg/day for children 2 to 5 years. Duration of treatment with eltrombopag varied per cohort (cohort 1: day 14 to six months; cohort 2: day 14 to three months; cohort 3: day one to six months). ATG and cyclosporine were administered as standard immunosuppression therapy[1].

The study's primary efficacy endpoint was hematologic complete response at six months defined by absolute neutrophil count >=1,000/Mu l, hemoglobin >=10 gm/dL, and platelets >=100,000/Mu l. Secondary endpoints included partial and overall hematologic responses at three months, six months, and yearly; survival; self-reported health outcomes; relapse, paroxysmal nocturnal hemoglobinuria (PNH) and clonal evolution as defined by a new clonal cytogenetic abnormality or characteristic dysplastic or leukemic changes in marrow consistent with myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML)[1].

About Eltrombopag
Eltrombopag, marketed as Promacta^® in the US and Revolade^® in countries outside the US, is approved in more than 100 countries worldwide for the treatment of thrombocytopenia in adult patients with chronic immune (idiopathic) thrombocytopenic purpura (ITP) who have had an inadequate response or are intolerant to other treatments, in over 45 countries worldwide for the treatment of patients with severe aplastic anemia (SAA) who are refractory to other treatments, and in more than 50 countries for the treatment of thrombocytopenia in patients with chronic hepatitis C to allow them to initiate and maintain interferon-based therapy. Eltrombopag is approved in the United States and in the European Union for the treatment of thrombocytopenia in pediatric patients 1 year and older with chronic immune (idiopathic) thrombocytopenia (ITP) who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy.